A glimmer of cure opens in neuroblastoma, the most frequent extracranial solid tumor of the pediatric age.
A treatment based on CAR-T cells, immune cells taken from patients and genetically modified to recognize cancer, has shown efficacy for the first time in the majority of patients suffering from the most severe form of the disease.
The treatment was developed by doctors and researchers from the Bambino Gesù Pediatric Hospital in Rome;
the data relating to the first trial conducted on 27 patients were illustrated in the New England Journal of Medicine.
The treatment proved to be safe and effective: 63% of patients had a response to the new treatment and more than half of them experienced a complete remission of the disease.
At 3 years, 60% of patients were alive;
half of them (36%) had no signs of ongoing disease.
Finally, CAR-T cells were present in the body 2-3 years after the infusion, supporting therapeutic efficacy over time.
"It is the first time at an international level that a study on the use of CAR-T against solid tumors has achieved such encouraging results and on such a large case series", commented the coordinator of the trial Franco Locatelli, head of the research area and clinical area of Oncohaematology, Cell Therapy, Gene Therapies and Hematopoietic Transplantation of the Child Jesus, as well as full professor of Pediatrics at the Catholic University of the Sacred Heart.
"We finally have one more therapeutic weapon that can be used to treat children diagnosed with neuroblastoma."
“Today we have the possibility of being able to use the same type of CAR-T cells also in different types of brain tumors.
We have already prepared a clinical protocol under evaluation for the final refinements at the Istituto Superiore di Sanità and, once we receive approval from the regulatory agencies, we will also begin a clinical trial in brain tumors in children and young adults up to 35 years of age. of age".
This is what Franco Locatelli, head of the research area and clinical area of Oncohematology, Cell Therapy, Gene Therapies and Hematopoietic Transplantation of the Child Jesus anticipated, during the presentation of the clinical study on the use of new CAR-T cells in children and boys with neuroblastoma.
The tumors subject to the new experiment, explained Locatelli, will be different brain tumors:
